According to Orphan Drug Designation program of the US Food and Drug Administration (FDA), orphan diseases are defined as rare diseases that affects less than 2,00,000 Americans. The global orphan drugs market is expected to benefit from patent extensions like Orphan Drug Exclusivity (ODE), increasing focus of vendors on orphan drug products, and financial incentives provided by government in form of tax credits and waived FDA fees. For instance, the FDA grants a seven-year patent exclusivity to orphan drugs to treat a rare disease. Therefore, any other drug with the same indication cannot be approved by the FDA during these seven-years period. In addition, they also provide tax credits up to 50% of the total R&D cost after the drug receives the orphan drug designation. This credit can be used by the sponsor during the R&D phase of any drug irrespective of the drug designation. The orphan designated drugs are also exempted from paying the FDA fees. Thus, these financial incentives and grants for orphan drug attract many vendors to invest in developing drugs for rare diseases and make huge profits.

Technavio’s analysts forecast the global orphan drugs market to grow at a CAGR of 10.20% during the period 2017-2021.

Covered in this report

The report covers the present scenario and the growth prospects of the global orphan drugs market for 2017-2021. To calculate the market size, the report presents a detailed picture of the market by way of study, synthesis, and summation of data from multiple sources.

The market is divided into the following segments based on geography:

Americas

APAC

EMEA

Technavio's report, Global Orphan Drugs Market 2017-2021, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of operating in this market.

Key vendors

AbbVie

Celgene

F. Hoffmann La Roche

Novartis

Other prominent vendors

Amgen

Actelion

Alexion Pharmaceuticals

Bayer

Biogen

Bristol-Myers Squibb

Eli Lilly

Johnson & Johnson

Novo Nordisk

Pfizer

Sanofi

Shire

Teva pharmaceutical

Market driver

Large growth opportunities through orphan drug designation.

For a full, detailed list, view our report

Market challenge

Costly treatment regimen results in financial burden on patients.

For a full, detailed list, view our report

Market trend

Emerging application for orphan drug development.

For a full, detailed list, view our report

Key questions answered in this report

What will the market size be in 2021 and what will the growth rate be?

What are the key market trends?

What is driving this market?

What are the challenges to market growth?

Who are in this market space?

What are the market opportunities and threats faced by the key vendors??

Commenting on the report, an analyst from Technavio’s team said: “One trend in market is repurposing of non-orphan drugs to orphan drugs. The orphan drugs market has witnessed many successful repositioning of drugs from non-orphan label to orphan label. Since the implementation of the orphan drug legislation in the United States, more than 69 drugs which were first approved by US FDA for the treatment of rare diseases were not entirely new and had been repurposed from the non-orphan label to the orphan label. Today, most of the pharmaceutical and biotechnological companies has adopted the strategy of drug repurposing in order to save time and money. Repurposing of an already approved drug for a different indication helps to mitigate the risk of drug failure as the drug has undergone the pharmacovigilance regulatory requirement and post-marketing survey. This reduces the risk of heavy financial loss to the manufacturer. In addition, the drug repurposing strategy also helps manufacturers to extend their product life cycle by getting orphan drug status, and also prevents their product from generic competition. Moreover, the external forces such as availability of huge amount of data, computational tools which helps in effective analysis of data and the advent of precession medicines for development of drugs that target a specific diseased population, has led to increase the adoption of this strategy.”

According to the report, one driver in market is high unmet need drives growth. The global orphan drugs market is growing at a rapid pace. Yet, it represents a high unmet need for the development of more efficacious drugs with fewer side-effects. Though the market is relatively small pertaining to the small patient pool, the return on investment is very high owing to the expensive orphan drugs. It has encouraged many big as well as small emerging pharmaceutical and biotechnological companies to invest in this market. The growing prevalence of rare diseases combined with the rising awareness among population and higher life expectancy rates are helping the global orphan drugs market to grow at a rapid rate. Additionally, government initiatives worldwide have positively influenced R&D on orphan drugs. Further, the number of marketing approvals of these drugs has shown a dramatic increase over the past few years.

Further, the report states that one challenge in market is costly treatment regimen results in financial burden on patients. The orphan drugs market is currently witnessing a growing concern due to increasing cost of orphan drugs and the need for a cost-effective therapy to treat rare disease in developing countries. The reason behind the rise in cost of these drugs is due to lack of competition and low density of drugs marketed for the treatment of same indication. The low availability of alternatives is because of the enforcement of patent exclusivity rule under the US ODA, which restricts the approval of an alternative drug until it represents evidence of being superior to the first-approved drug in terms of safety and efficacy. For instance, the average cost of a rare disease therapy for a year is more than the cost of a car. This limits the access to orphan drug for patients living in developing countries such as India and China. As the reimbursement rates for these drugs are low, healthcare professionals hesitate to prescribe these drugs considering the financial status of a patient.